Catabasis Pharmaceuticals and Sarepta Therapeutics Announce a Joint Research Collaboration in Duchenne Muscular Dystrophy
29 September 2016 - 10:00PM
Business Wire
Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage
biopharmaceutical company (“Catabasis”), and Sarepta Therapeutics,
Inc. (NASDAQ:SRPT), a commercial-stage developer of innovative
RNA-targeted therapeutics (“Sarepta”), today announced a joint
research collaboration to explore a combination drug treatment
approach for Duchenne muscular dystrophy (DMD). The two companies
will contribute their respective expertise to study an exon
skipping treatment developed by Sarepta, together with an oral
NF-kB inhibition treatment developed by Catabasis in a mouse model
of DMD.
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“We are excited to work with Sarepta on this joint research
collaboration, which to our knowledge is the first time two
companies are testing a combination of investigational therapies to
treat Duchenne. Although we believe edasalonexent (CAT-1004) has
the potential to be a disease-modifying monotherapy, we think there
is benefit to exploring innovative ways to make the most meaningful
difference in this devastating disease”, said Jill C. Milne, Ph.D.,
chief executive officer of Catabasis. “In addition to our continued
development of edasalonexent, we are pleased to take the first step
via this collaboration to determine if edasalonexent may be
complementary to an exon-skipping treatment strategy in the
treatment of DMD using a preclinical model.”
“We recognize the extreme unmet medical need in DMD and are
committed to determining the best treatment strategies for patients
affected by Duchenne,” said Edward Kaye, M.D., Sarepta’s chief
executive officer. “We believe exon skipping has the potential to
target the underlying genetic cause of the disease by restoring the
mRNA reading frame to produce dystrophin in skeletal muscle. We are
pleased to initiate activities with Catabasis to evaluate a
potential combination treatment approach of exon-skipping and NF-kB
inhibition in DMD.”
NF-kB inhibition and exon-skipping represent two novel
investigational treatment strategies in Duchenne, each with the
potential for disease-modifying effects when used as monotherapy.
The objective of the joint research is to study the safety and
efficacy of combining these two treatment strategies using a mouse
model of DMD, including evaluating the potential for additional or
synergistic benefits.
About CatabasisAt Catabasis Pharmaceuticals, our mission
is to bring hope and life-changing therapies to patients and their
families. Our SMART (Safely Metabolized And Rationally Targeted)
linker drug discovery platform enables us to engineer molecules
that simultaneously modulate multiple targets in a disease. We are
applying our SMART linker platform to build an internal pipeline of
product candidates for rare diseases and plan to pursue
partnerships to develop additional product candidates. For more
information on the Company's drug discovery platform and pipeline
of drug candidates, please visit www.catabasis.com.
About Edasalonexent (CAT-1004)Edasalonexent (CAT-1004) is
an oral small molecule that has the potential to be a
disease-modifying therapy for all patients affected by Duchenne
muscular dystrophy (DMD or Duchenne), regardless of the underlying
mutation. Edasalonexent inhibits NF-kB, a protein that is activated
in Duchenne and drives inflammation and fibrosis, muscle
degeneration and suppresses muscle regeneration. In animal models
of DMD, edasalonexent inhibited NF-kB, reduced muscle degeneration
and improved muscle regeneration and function, and beneficial
effects were observed in skeletal, diaphragm and cardiac muscle.
The FDA has granted orphan drug, fast track and rare pediatric
disease designations and the European Commission has granted orphan
medicinal product designation to edasalonexent for the treatment of
DMD. We have previously reported safety, tolerability and reduction
in NF-kB activity in Phase 1 trials in adults. We are currently
conducting the MoveDMD® trial of edasalonexent in 4-7 year-old boys
affected by Duchenne. From Part A of the MoveDMD trial, we have
reported that edasalonexent was generally well tolerated with no
safety signals observed and we observed successful NF-kB target
engagement. Pharmacokinetic results demonstrated edasalonexent
plasma exposure levels consistent with those previously observed in
adults at which inhibition of NF-kB was observed.
About Sarepta TherapeuticsSarepta
Therapeutics is a commercial-stage biopharmaceutical company
focused on the discovery and development of unique RNA-targeted
therapeutics for the treatment of rare neuromuscular diseases. The
Company is primarily focused on rapidly advancing the development
of its potentially disease-modifying DMD drug candidates, including
EXONDYS 51, designed to skip exon 51 and approved under the
accelerated approval pathway. For more information, please visit us
at www.sarepta.com.
About Duchenne Muscular DystrophyDMD is an X-linked rare
degenerative neuromuscular disorder causing severe progressive
muscle loss and premature death. One of the most common fatal
genetic disorders, DMD affects approximately one in every
3,500-5,000 males worldwide. A devastating and incurable
muscle-wasting disease, DMD is associated with specific errors in
the gene that codes for dystrophin, a protein that plays a key
structural role in muscle fiber function. Progressive muscle
weakness in the lower limbs spreads to the arms, neck and other
areas. Eventually, increasing difficulty in breathing due to
respiratory muscle dysfunction requires ventilation support, and
cardiac dysfunction can lead to heart failure. The condition is
universally fatal, and death usually occurs before the age of
30.
Forward-Looking StatementsAny statements in this press
release about future expectations, plans and prospects for
Catabasis and/or Sarepta, including statements about future
clinical trial plans and other statements containing the words
“believes,” “anticipates,” “plans,” “expects,” “may” and similar
expressions, constitute forward-looking statements within the
meaning of the Private Securities Litigation Reform Act of 1995.
These forward-looking statements include statements regarding the
joint research collaboration plans of Sarepta and Catabasis to
explore a combination drug treatment approach for DMD and the
potential benefit of the products being researched in DMD. Actual
results may differ materially from those indicated by such
forward-looking statements as a result of various important
factors, including: uncertainties inherent in the initiation and
completion of preclinical studies and clinical trials and clinical
development of Catabasis’ or Sarepta’s product candidates;
availability and timing of results from preclinical studies and
clinical trials; whether interim results from a clinical trial will
be predictive of the final results of the trial or the results of
future trials; expectations for regulatory approvals to conduct
trials or to market products; availability of funding sufficient
for Catabasis’ or Sarepta’s foreseeable and unforeseeable operating
expenses and capital expenditure requirements; other matters that
could affect the availability or commercial potential of Catabasis’
or Sarepta’s product candidates; and general economic and market
conditions and other factors discussed in the “Risk Factors”
section of each of Catabasis’ and Sarepta’s Quarterly Report on
Form 10-Q for the period ended June 30, 2016, which are each on
file with the Securities and Exchange Commission, and in other
filings that Catabasis or Sarepta may make with the Securities and
Exchange Commission in the future. In addition, the forward-looking
statements included in this press release represent Catabasis’ and
Sarepta’s views as of the date of this press release. Each of
Catabasis and Sarepta anticipates that subsequent events and
developments will cause their respective views to change. However,
while either Catabasis or Sarepta may elect to update these
forward-looking statements at some point in the future, each
company specifically disclaims any obligation to do so. These
forward-looking statements should not be relied upon as
representing the views of either Catabasis or Sarepta as of any
date subsequent to the date of this release.
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Catabasis Investor and Media
Contact:Catabasis Pharmaceuticals, Inc.Andrea Matthews,
617-349-1971amatthews@catabasis.comorSarepta
Therapeutics, Inc.Media and Investors:Ian Estepan,
617-274-4052iestepan@sarepta.comorW2O GroupBrian Reid,
212-257-6725breid@w2ogroup.com
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